In recent first study testing, scientists are testing a one-time treatment which permanently alters DNA in blood cells which suggests a treatment and possible cure for sickle cell disease and beta thalassem (USNews, 2020). In this treatment, stem cells are removed from a patient's blood, and a tool called CRISPR aims to remove the switching gene that defects hemoglobin. Then, patients are given strong medicines to kill off other, flawed blood-producing cells, and the stem cells are given back (USNews, 2020). So far, the gene editing tests suggest it is working as there are desired, encouraging effects (USNews, 2020).
It was so interesting to read this because I personally know someone who struggles with this disease. I hope this is turns out to be a successful research!ReplyDelete